艾美赛珠单抗治疗伴有同种抗体的 3 型血管性血友病患儿的疗效。
Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies.
机构信息
Division of Hematology and Oncology, Department of Pediatrics, University of Michigan, Ann Arbor, MI.
Blood Research Institute, Blood Center of Wisconsin, Milwaukee, WI.
出版信息
Blood Adv. 2019 Sep 24;3(18):2748-2750. doi: 10.1182/bloodadvances.2019000656.
Abstract
Type 3 von Willebrand disease with alloantibodies is a rare clinical entity with few treatment options. Emicizumab prophylaxis in such patients may result in improved hemarthrosis control, lower cost, and enhanced quality of life.
摘要
3 型血管性血友病伴同种抗体是一种罕见的临床病症，治疗选择有限。此类患者接受emicizumab 预防治疗可能会改善关节积血控制、降低成本并提高生活质量。
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